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Gene Therapy Shows Lasting Benefit for Children With Rare Disorder

For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational ...
New Atlas

95% of kids with “bubble boy” disease cured by one-time gene therapy

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated ...
Just-Food

USA: Naturally Vitamins extends contract for US distribution Of Wobenzym(R)

Phoenix-based Marlyn Nutraceuticals, dba Naturally Vitamins, has extended its contract with MUCOS Pharma of Germany giving them exclusive rights to distribute Wobenzym in the US. Wobenzym, which will ...
BioWorld

Autologous B-cell therapy WFX-001 corrects systemic α-GAL enzyme deficiency in vivo

BioWorld - Wednesday, August 21, 2024 Breaking News: BioWorld celebrates nine wins at 2024 APEX Awards See today's BioWorld Science Home » Autologous B-cell therapy WFX-001 corrects systemic α-GAL ...
Business Wire

GC Pharma Announces EMA Grants Orphan Drug Designation to Hunterase ICV, The World’s First Enzyme Replacement Therapy for Mucopolysaccharidosis Type II Administered by ICV ...

YONGIN, South Korea--(BUSINESS WIRE)--GC Pharma (006280.KS) today announced that the European Medicines Agency (EMA) granted orphan drug designation (ODD) to Hunterase ICV (intracerebroventricular, ...
Medscape

Radiotherapy Defers Systemic Therapy in Oligometastatic RCC

Serial metastasis-directed radiotherapy extended time off systemic therapy while maintaining favorable progression-free and overall survival in patients with oligometastatic clear-cell renal cell ...